Drug delivery to the brain is a major medical challenge that prevents treatment of many brain disorders, including the highly aggressive and deadly brain cancer Glioblastoma (GBM). This demands innovative approaches for drug delivery to GBM be developed. We previously established that therapeutically “armed” neural stem cells (NSCs) are advantageous over traditional delivery methods due to: 1) tumor-selective homing to primary and invasive GBM deposits, and 2) continuous long-term delivery of therapeutics directly to GBM. NSC-based therapy could effectively treat GBM and save lives, but limitations in isolation and patient-specificity currently prevents their clinical advancement.

We propose to progress NSC-based therapy for GBM by applying transformative advances in cellular reprogramming technology. In this proposal, we will develop and critically assess a novel type of NSC as cellular vehicles for GBM treatment. By deriving NSCs from fibroblasts, this cellular reprogramming technology provides a model for the eventual generation of personalized patient-specific NSCs to recognize the killing of aggressive GBMs.